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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Therapy Information
Study Record Dates
Participation Criteria
Locations
Regulatory Information
Resources/Links
Summary
Safety and Tolerability of Intravitreal Administration of VG901 in Patients With Retinitis Pigmentosa Due to Mutations in the CNGA1 Gene
NCTID
NCT06291935
(View at clinicaltrials.gov)
Description
The goal of this phase 1 clinical trial is to learn about the safety and efficacy of a gene therapy, VG901, in patients with a rare disorder of the eye called Retinitis Pigmentosa. The main questions the study aims to answer are: * What is the best tolerated dose and are there any side effects, in particular any inflammatory reactions post drug administration? * Are there any early signs of efficacy on visual function? Participants will be administered a single intravitreal dose of VG901 into the most affected eye through a syringe and followed up for a year to monitor safety and efficacy. There will be two cohorts of participants in this study. Study Cohort 1 will receive the low dose and Study Cohort 2 will receive the high dose as specified in the Protocol.
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Development Status
Active
Indication
Retinitis Pigmentosa
Disease Ontology Term
DOID:110007
Compound Name
VG901
Compound Description
AAV2.NN-CNGA1
Sponsor
ViGeneron GmbH
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
6
Results Posted
Not Available
Therapy Information
Target Gene/Variant
CNGA1
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Intravitreal
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
Undisclosed low dose
Dose 2
Undisclosed high dose
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1
Submit Date
2024-02-01
Completion Date
2026-04
Last Update
2025-03-28
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
1
Locations
Germany
Regulatory Information
Has US IND
False
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates
First patient dosed 4/10/24
Resources/Links
News and Press Releases
ViGeneron Announces First Patient Dosed in Phase 1b Clinical Trial of VG901 For the Intravitreal Treatment of Retinitis Pigmentosa
ViGeneron Announces FDA Rare Pediatric Disease Designation for VG901 and DSMB Approval to Advance Dose Escalation in Phase 1b Retinitis Pigmentosa Trial
Preclinical Publications
Gene therapy restores vision and delays degeneration in the CNGB1(-/-) mouse model of retinitis pigmentosa
Gene therapy restores vision and delays degeneration in the CNGB1(-/-) mouse model of retinitis pigmentosa